Gene therapy Archives

News and Trends 23 Jun 2023

Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy

Sarepta Therapeutics, Inc. has announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an Adeno-associated virus (AAV)-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval […]

June 23, 2023 - 3 minutesmins - By Jim Cornall

In Depth 19 Jun 2023

Gene therapy: a promising solution for sickle cell disease

Sickle cell disease is debilitating, and many people who suffer with it find that their lives are defined by pain. This means a curative treatment would be completely life–altering, and, as we observe World Sickle Cell Day on June 19, we explore how gene therapy could be the most promising solution for curing sickle cell […]

June 19, 2023 - 6 minutesmins - By Willow Shah-Neville

Beyond Biotech podcast gene cell therapy

Podcast 16 Jun 2023

Beyond Biotech podcast 50

The International Society for Cell & Gene Therapy (ISCT), the global society of more than 3,000 members dedicated to the translation of cell and gene therapies (CGT) into safe and effective treatments to improve patient lives, recently hosted the largest translation-focused event connecting academic, regulatory and industry experts from around the world to collaborate and […]

June 16, 2023

News and Trends 8 Jun 2023

Kate Therapeutics launches with $51M to develop genetic medicines for muscle and heart diseases

Gene therapy company Kate Therapeutics Inc. (KateTx), has emerged from stealth mode with a $51 million Series A financing. In addition, the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM). There are a large number of genetically defined and complex muscle […]

June 8, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 7 Jun 2023

AAVantgarde closes €61M financing to move gene delivery program to the clinic

AAVantgarde Bio, a clinical-stage, Italian-based international biotechnology company with two proprietary adeno-associated viral (AAV) vector platforms for large gene delivery, has closed a €61 million ($65.1 million) Series A financing. The amount represents the largest series A round ever in Italian biotech. AAVantgarde has two proprietary, AAV-based large gene delivery platforms: one leveraging DNA recombination, […]

June 7, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 1 Jun 2023

ElevateBio raises $401M for cell and gene therapies

ElevateBio, LLC (ElevateBio), a technology-driven company focused on cell and gene therapies, has closed its $401 million Series D financing. “We have made significant strides in scaling our technologies and end-to-end capabilities in our pursuit to become the world’s most indispensable cell and gene therapy technology company. We are thrilled to welcome Khalil to our […]

June 1, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 26 May 2023

Novartis buys AVROBIO cystinosis gene therapy program for $87.5M

AVROBIO, Inc., a clinical-stage gene therapy company working to free people from a lifetime of genetic disease, has agreed to sell its investigational hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis to Novartis for $87.5 million in cash. AVROBIO retains full rights to its portfolio of first-in-class HSC gene therapies for […]

May 26, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 26 May 2023

Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

Rocket Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the company’s investigational lentiviral-based gene therapy for pyruvate kinase deficiency (PKD). PKD is a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. RMAT designation was granted based […]

May 26, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 22 May 2023

FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

The U.S. Food and Drug Administration has approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. The approval of Vyjuvek was granted to […]

May 22, 2023 - 3 minutesmins - By Jim Cornall

In Depth 19 May 2023

Can cardiovascular diseases be cured? The future of heart treatment

Cardiovascular diseases, characterized as a group of disorders of the heart and blood vessels, are the leading cause of death worldwide. In fact, in the U.S. alone, it is thought that one person dies every 33 seconds from cardiovascular disease. Although clearly a huge health problem throughout the world, developing cures for cardiovascular diseases is […]

May 19, 2023 - 11 minutesmins - By Willow Shah-Neville

News and Trends 18 May 2023

8 rare diseases selected for gene therapy trial portfolio

The Foundation for the National Institutes of Health (FNIH) says the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. This portfolio pioneers a novel approach to gene therapy development for rare diseases by demonstrating that manufacturing and testing standards can provide a streamlined approval […]

May 18, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 18 May 2023

Sania Therapeutics launches to advance neural circuit dysfunction medicines

Sania Therapeutics, which develops genetic medicines for neural circuit dysfunction, has launched with the unveiling of its platforms at the American Society of Gene & Cell Therapy (ASGCT) conference. Sania Therapeutics’ approach combines the evolution of adeno-associated viruses (AAV) in human neural circuits, the fundamental components of the nervous system, and tuneable conditionally-activated ion channels. […]

May 18, 2023 - 2 minutesmins - By Jim Cornall

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Gene therapy

Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy

Gene therapy: a promising solution for sickle cell disease

Beyond Biotech podcast 50

Kate Therapeutics launches with $51M to develop genetic medicines for muscle and heart diseases

AAVantgarde closes €61M financing to move gene delivery program to the clinic

ElevateBio raises $401M for cell and gene therapies

Novartis buys AVROBIO cystinosis gene therapy program for $87.5M

Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

Can cardiovascular diseases be cured? The future of heart treatment

8 rare diseases selected for gene therapy trial portfolio

Sania Therapeutics launches to advance neural circuit dysfunction medicines

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Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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